Evidence registry
- Evidence reviewed through
- Aug 11, 2026
About this page
Official trial records are the source for study status, eligibility, and design. Peer-reviewed publications are the source for scientific details and reported results.
“Not publicly disclosed” means no program-specific public source reporting that detail was found in the sources reviewed. Read the evidence methodology.
DB-OTO (CHORD)NCT05788536 · 4 linked sources · reviewed Jul 24, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | A multicenter study evaluating a one-time intracochlear OTOF gene therapy in children and infants with congenital hearing loss caused by biallelic OTOF variants. | |
| Sponsor | Regeneron Pharmaceuticals | |
| Target gene | OTOF | |
| Strategy | Gene replacement | |
| Vector / payload | Dual AAV1 vectors reconstituting full-length human OTOF transcript variant 5 (hOTOFv5) | |
| Promoter | mMyo15, a synthetic hair-cell-specific promoter derived from murine Myosin 15a regulatory sequence | |
| Phase | Phase 1/2 | |
| Status | Actively Recruiting | |
| FDA approval | Yes | |
| Population | Children and infants with biallelic OTOF-related hearing loss | |
| Age group | Pediatric | |
| Age range | Up to 17 years; cohort-specific | |
| Study start date | Jun 27, 2023 | |
| Administration | One-time intracochlear injection through the round-window membrane with lateral semicircular canal fenestration | |
| Cochlear access | Round-window membrane injection with lateral semicircular canal fenestration | |
| Fenestration | Yes — lateral semicircular canal (LSCC) | |
| Laterality | Unilateral dose escalation; bilateral expansion | |
| Geography | Germany, Japan, Spain, United Kingdom, and United States | |
| Enrollment | 30 | |
| Registry ID | NCT05788536 |
- Program summary
- A multicenter study evaluating a one-time intracochlear OTOF gene therapy in children and infants with congenital hearing loss caused by biallelic OTOF variants.
- Supporting sources for Program summary:
- Target gene
- OTOF
- Supporting sources for Target gene:
- Strategy
- Gene replacement
- Supporting sources for Strategy:
- Vector / payload
- Dual AAV1 vectors reconstituting full-length human OTOF transcript variant 5 (hOTOFv5)
- Supporting sources for Vector / payload:
- Promoter
- mMyo15, a synthetic hair-cell-specific promoter derived from murine Myosin 15a regulatory sequence
- Supporting sources for Promoter:
- Population
- Children and infants with biallelic OTOF-related hearing loss
- Supporting sources for Population:
- Age range
- Up to 17 years; cohort-specific
- Supporting sources for Age range:
- Study start date
- Jun 27, 2023
- Supporting sources for Study start date:
- Administration
- One-time intracochlear injection through the round-window membrane with lateral semicircular canal fenestration
- Supporting sources for Administration:
- Cochlear access
- Round-window membrane injection with lateral semicircular canal fenestration
- Supporting sources for Cochlear access:
- Fenestration
- Yes — lateral semicircular canal (LSCC)
- Supporting sources for Fenestration:
- Laterality
- Unilateral dose escalation; bilateral expansion
- Supporting sources for Laterality:
- Geography
- Germany, Japan, Spain, United Kingdom, and United States
- Supporting sources for Geography:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. A Study of DB-OTO Gene Therapy in Children/Infants With Hearing Loss Due to Otoferlin Mutations (CHORD), NCT05788536.
Official ClinicalTrials.gov record · Program-specific
- DB-OTO Gene Therapy for Inherited Deafness. New England Journal of Medicine. 2026. doi:10.1056/NEJMoa2400521.
Peer-reviewed study in people · Program-specific
- Functional, sustained recovery of hearing in Otoferlin-deficient mice using DB-OTO, a hair-cell-specific AAV-based gene therapy. Molecular Therapy Methods & Clinical Development. 2025. doi:10.1016/j.omtm.2025.101577.
Peer-reviewed laboratory or animal study · Program-specific
- U.S. Food and Drug Administration. OTARMENI (lunsotogene parvec-cwha), STN 125874. Approved April 23, 2026.
Official FDA record · Program-specific
AK-OTOF-101NCT05821959 · 4 linked sources · reviewed Jul 24, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | A dose-escalation and bilateral-expansion study of a dual-AAVAnc80 OTOF construct delivered with a dedicated intracochlear administration system. | |
| Sponsor | Akouos, Inc. / Eli Lilly | |
| Target gene | OTOF | |
| Strategy | Gene replacement | |
| Vector / payload | Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA | |
| Promoter | Ubiquitous promoter; specific identity is not publicly disclosed | |
| Phase | Phase 1/2 | |
| Status | Actively Recruiting | |
| FDA approval | No | |
| Population | People with OTOF-mediated sensorineural hearing loss | |
| Age group | Pediatric and adult | |
| Age range | Cohort 1a: 7–17 years Cohort 1b: first 3 participants are 2 years and older, next 3 may be any age Cohort 2: first 3 participants are 2 years and older, next 3 may be any age bilateral expansion: any age | |
| Study start date | Sep 15, 2023 | |
| Administration | One-time intracochlear administration using the Akouos delivery device | |
| Cochlear access | Transcanal tympanotomy to the round-window membrane with a stapes-footplate vent | |
| Fenestration | Yes — stapes footplate vent (fenestration) | |
| Laterality | Unilateral cohorts; bilateral expansion | |
| Geography | Canada, Taiwan, United Kingdom, and United States | |
| Enrollment | 22 | |
| Registry ID | NCT05821959 |
- Program summary
- A dose-escalation and bilateral-expansion study of a dual-AAVAnc80 OTOF construct delivered with a dedicated intracochlear administration system.
- Supporting sources for Program summary:
- Target gene
- OTOF
- Supporting sources for Target gene:
- Strategy
- Gene replacement
- Supporting sources for Strategy:
- Vector / payload
- Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA
- Supporting sources for Vector / payload:
- Promoter
- Ubiquitous promoter; specific identity is not publicly disclosed
- Supporting sources for Promoter:
- Population
- People with OTOF-mediated sensorineural hearing loss
- Supporting sources for Population:
- Age range
- Cohort 1a: 7–17 years Cohort 1b: first 3 participants are 2 years and older, next 3 may be any age Cohort 2: first 3 participants are 2 years and older, next 3 may be any age bilateral expansion: any age
- Supporting sources for Age range:
- Study start date
- Sep 15, 2023
- Supporting sources for Study start date:
- Administration
- One-time intracochlear administration using the Akouos delivery device
- Supporting sources for Administration:
- Cochlear access
- Transcanal tympanotomy to the round-window membrane with a stapes-footplate vent
- Supporting sources for Cochlear access:
- Fenestration
- Yes — stapes footplate vent (fenestration)
- Supporting sources for Fenestration:
- Laterality
- Unilateral cohorts; bilateral expansion
- Supporting sources for Laterality:
- Geography
- Canada, Taiwan, United Kingdom, and United States
- Supporting sources for Geography:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. A Study of AK-OTOF Gene Therapy in Participants With OTOF-Mediated Hearing Loss, NCT05821959.
Official ClinicalTrials.gov record · Program-specific
- Preliminary safety and efficacy of AK-OTOF gene therapy for OTOF-mediated hearing loss. Genetics in Medicine Open. 2026. doi:10.1016/j.gimo.2026.103685.
Conference abstract · Program-specific
- Akouos. Preclinical Development of a Genetic Medicine for Otoferlin Gene-mediated Hearing Loss: AK-OTOF. Technical presentation.
Sponsor technical source · Program-specific
- U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Licensed-product list reviewed July 23, 2026.
Official FDA record · Background only
SENS-501 (Audiogene)NCT06370351 · 4 linked sources · reviewed Aug 11, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | An adaptive dose-escalation and expansion study evaluating unilateral intracochlear SENS-501 administration in very young children with OTOF-related hearing loss. | |
| Sponsor | Sensorion | |
| Target gene | OTOF | |
| Strategy | Gene replacement | |
| Vector / payload | Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution | |
| Promoter | CMV promoter | |
| Phase | Phase 1/2 | |
| Status | Active, Not Recruiting | |
| FDA approval | No | |
| Population | Children with severe-to-profound pre-lingual OTOF-related hearing loss | |
| Age group | Pediatric | |
| Age range | 6–31 months | |
| Study start date | Jun 21, 2024 | |
| Administration | Unilateral intracochlear injection with a proprietary delivery device | |
| Cochlear access | Round-window membrane injection; translational work describes stapedotomy pressure relief | |
| Fenestration | Yes — stapes footplate (stapedotomy) | |
| Laterality | Unilateral | |
| Geography | Australia and France | |
| Enrollment | 12 | |
| Registry ID | NCT06370351 |
- Program summary
- An adaptive dose-escalation and expansion study evaluating unilateral intracochlear SENS-501 administration in very young children with OTOF-related hearing loss.
- Supporting sources for Program summary:
- Target gene
- OTOF
- Supporting sources for Target gene:
- Strategy
- Gene replacement
- Supporting sources for Strategy:
- Vector / payload
- Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution
- Supporting sources for Vector / payload:
- Population
- Children with severe-to-profound pre-lingual OTOF-related hearing loss
- Supporting sources for Population:
- Study start date
- Jun 21, 2024
- Supporting sources for Study start date:
- Administration
- Unilateral intracochlear injection with a proprietary delivery device
- Supporting sources for Administration:
- Cochlear access
- Round-window membrane injection; translational work describes stapedotomy pressure relief
- Supporting sources for Cochlear access:
- Fenestration
- Yes — stapes footplate (stapedotomy)
- Supporting sources for Fenestration:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. Audiogene: Gene Therapy Trial for Children With OTOF-Related Hearing Loss, NCT06370351.
Official ClinicalTrials.gov record · Program-specific
- Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness. Molecular Therapy Advances. 2026. doi:10.1016/j.omta.2026.201762.
Peer-reviewed laboratory or animal study · Program-specific
- Sensorion. SENS-501 Dedicated Surgical Approach for Gene Therapy. Corporate presentation. 2024.
Sponsor technical source · Program-specific
- U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Licensed-product list reviewed July 23, 2026.
Official FDA record · Background only
OTOV101N + OTOV101CNCT05901480 · 4 linked sources · reviewed Jul 24, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | An investigator-initiated program evaluating a dual-vector OTOF gene-replacement approach in people with DFNB9 hearing loss. | |
| Sponsor | Otovia Therapeutics | |
| Target gene | OTOF | |
| Strategy | Gene replacement | |
| Vector / payload | Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution | |
| Promoter | mMyo15 hair-cell-specific promoter | |
| Phase | Early clinical | |
| Status | Active | |
| FDA approval | No | |
| Population | People aged 1 year and older with OTOF-related hearing loss | |
| Age group | Pediatric and adult | |
| Age range | 1 year and older | |
| Study start date | Jun 26, 2023 | |
| Administration | Intracochlear administration | |
| Cochlear access | Round-window membrane injection; preclinical program work used a transmastoid facial-recess corridor | |
| Fenestration | No separate fenestration reported; round-window membrane injection | |
| Laterality | Cohort-dependent | |
| Geography | China | |
| Enrollment | Not publicly disclosed | |
| Registry ID | NCT05901480 |
- Program summary
- An investigator-initiated program evaluating a dual-vector OTOF gene-replacement approach in people with DFNB9 hearing loss.
- Supporting sources for Program summary:
- Target gene
- OTOF
- Supporting sources for Target gene:
- Strategy
- Gene replacement
- Supporting sources for Strategy:
- Vector / payload
- Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution
- Supporting sources for Vector / payload:
- Promoter
- mMyo15 hair-cell-specific promoter
- Supporting sources for Promoter:
- Population
- People aged 1 year and older with OTOF-related hearing loss
- Supporting sources for Population:
- Study start date
- Jun 26, 2023
- Supporting sources for Study start date:
- Administration
- Intracochlear administration
- Supporting sources for Administration:
- Cochlear access
- Round-window membrane injection; preclinical program work used a transmastoid facial-recess corridor
- Supporting sources for Cochlear access:
- Fenestration
- No separate fenestration reported; round-window membrane injection
- Supporting sources for Fenestration:
- Enrollment
- Not publicly disclosed
- Supporting sources for Enrollment:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. OTOF Gene Therapy for Patients With DFNB9, NCT05901480.
Official ClinicalTrials.gov record · Program-specific
- AAV gene therapy for autosomal recessive deafness 9: a single-arm trial. Nature Medicine. 2025. doi:10.1038/s41591-025-03773-w.
Peer-reviewed study in people · Program-specific
- Preclinical Efficacy and Safety Evaluation of AAV-OTOF in DFNB9 Mouse Model and Nonhuman Primate. Advanced Science. 2024. doi:10.1002/advs.202306201.
Peer-reviewed laboratory or animal study · Program-specific
- U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Licensed-product list reviewed July 23, 2026.
Official FDA record · Background only
EH002NCT06722170 · 3 linked sources · reviewed Jul 24, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | A dose-escalation study of EH002 administered into one or both ears for congenital hearing loss caused by OTOF variants. | |
| Sponsor | Yilai Shu / Eye & ENT Hospital of Fudan University | |
| Target gene | OTOF | |
| Strategy | Gene replacement | |
| Vector / payload | OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed | |
| Promoter | Not publicly disclosed | |
| Phase | Early clinical | |
| Status | Actively Recruiting | |
| FDA approval | No | |
| Population | People with DFNB9 congenital hearing loss | |
| Age group | Pediatric and adult | |
| Age range | 6 months and older; additional eligibility criteria apply | |
| Study start date | Nov 22, 2024 | |
| Administration | Intracochlear injection | |
| Cochlear access | The precise cochlear access route is not publicly disclosed | |
| Fenestration | Not publicly disclosed | |
| Laterality | One or both ears; one or two administrations | |
| Geography | China | |
| Enrollment | 24 | |
| Registry ID | NCT06722170 |
- Program summary
- A dose-escalation study of EH002 administered into one or both ears for congenital hearing loss caused by OTOF variants.
- Supporting sources for Program summary:
- Sponsor
- Yilai Shu / Eye & ENT Hospital of Fudan University
- Supporting sources for Sponsor:
- Vector / payload
- OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed
- Supporting sources for Vector / payload:
- Promoter
- Not publicly disclosed
- Supporting sources for Promoter:
- Population
- People with DFNB9 congenital hearing loss
- Supporting sources for Population:
- Age range
- 6 months and older; additional eligibility criteria apply
- Supporting sources for Age range:
- Study start date
- Nov 22, 2024
- Supporting sources for Study start date:
- Administration
- Intracochlear injection
- Supporting sources for Administration:
- Cochlear access
- The precise cochlear access route is not publicly disclosed
- Supporting sources for Cochlear access:
- Fenestration
- Not publicly disclosed
- Supporting sources for Fenestration:
- Laterality
- One or both ears; one or two administrations
- Supporting sources for Laterality:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. EH002 in Patients With DFNB9 Congenital Hearing Loss, NCT06722170.
Official ClinicalTrials.gov record · Program-specific
- AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. The Lancet. 2024. doi:10.1016/S0140-6736(23)02874-X.
Peer-reviewed study in people · Background only
- U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Licensed-product list reviewed July 23, 2026.
Official FDA record · Background only
SKY-GJB2 (SONIX)NCT07627971 · 5 linked sources · reviewed Jul 27, 2026
| Field | Information shown | Supporting source |
|---|---|---|
| Program summary | A first-in-human pediatric study of an AAV GJB2 gene-replacement therapy delivered once to one cochlea using the SKY-CAT investigational delivery system. | |
| Sponsor | Skylark Bio Inc. | |
| Target gene | GJB2 | |
| Strategy | Gene replacement | |
| Vector / payload | AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed | |
| Promoter | Not publicly disclosed | |
| Phase | Phase 1/2 | |
| Status | Actively Recruiting | |
| FDA approval | No | |
| Population | Children with biallelic GJB2-mediated hearing loss | |
| Age group | Pediatric | |
| Age range | 9 months–7 years | |
| Study start date | May 28, 2026 | |
| Administration | Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT | |
| Cochlear access | Round-window access via transcanal endoscopic tympanotomy | |
| Fenestration | No additional fenestration reported | |
| Laterality | Unilateral | |
| Geography | United States | |
| Enrollment | 10 | |
| Registry ID | NCT07627971 |
- Program summary
- A first-in-human pediatric study of an AAV GJB2 gene-replacement therapy delivered once to one cochlea using the SKY-CAT investigational delivery system.
- Supporting sources for Program summary:
- Target gene
- GJB2
- Supporting sources for Target gene:
- Strategy
- Gene replacement
- Supporting sources for Strategy:
- Vector / payload
- AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed
- Supporting sources for Vector / payload:
- Promoter
- Not publicly disclosed
- Supporting sources for Promoter:
- Population
- Children with biallelic GJB2-mediated hearing loss
- Supporting sources for Population:
- Study start date
- May 28, 2026
- Supporting sources for Study start date:
- Administration
- Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT
- Supporting sources for Administration:
- Cochlear access
- Round-window access via transcanal endoscopic tympanotomy
- Supporting sources for Cochlear access:
- Fenestration
- No additional fenestration reported
- Supporting sources for Fenestration:
Full source records
These citations correspond to the source labels attached to individual facts above.
- ClinicalTrials.gov. SONIX: A Study of SKY-GJB2 in Pediatric Participants With GJB2-Mediated Hearing Loss, NCT07627971.
Official ClinicalTrials.gov record · Program-specific
- Skylark Bio and Forge Biologics announce AAV development and manufacturing partnership supporting SKY-GJB2 clinical development.
Sponsor technical source · Program-specific
- Skylark Bio. SONIX Study: SKY-GJB2-001 study overview and dosing description.
Sponsor technical source · Program-specific
- Lehigh Valley Health Network. SKY-GJB2-001 clinical trial description.
Participating-site trial page · Program-specific
- U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Licensed-product list reviewed July 23, 2026.
Official FDA record · Background only